The 21st Century Cures Act, recently signed into law by President Barack Obama, includes provisions that aim to speed up the process by which the U.S. Food and Drug Administration (FDA) reviews stem cell treatments for patients with degenerative diseases, such as multiple sclerosis, rheumatoid arthritis, and Parkinson’s disease.
This act also brings new dollars ($4.8 billion over 10 years) to the National Institutes of Health, partly with a goal of advancing biomedicine and regenerative therapies.
In a statement, Robert M. Califf, the FDA commissioner welcomed the act for establishing “a new program for the development of regenerative medicine products, an important and exciting new field that deserves this special focus.” Key elements in its provisions, Califf said, were an “enhance[d] … exchange of information among FDA, researchers and developers,” and the creation of a “research network and a public-private partnership to assist developers in generating definitive evidence” that could lead to quicker approval of therapies showing that they “indeed provide clinical benefits that are hoped for.”
The FDA will receive $500 million over 10 years to implement these programs.
Under the Cures Act, “regenerative medicine and advanced therapies” are defined so as to include gene therapy, gene-modified cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products that use these therapies or products, according to an “explainer” on the new law released by the Regulatory Affairs Professionals Society or RAPS.
The law was also welcomed by those working in regenerative therapies, including Dr. Steven Brody, chief scientific officer of StemGenex. At a mid-September FDA public hearing into the use of human cells, tissues and tissue-based products — in advance of an awaited agency guideline on such use — Brody emphasized the importance of a regulatory pathway that secures patient safety, while also accelerating the review of new regenerative treatments, such as stem cell therapies.
“Adult stem cells are potentially the most diverse and effective healing agents ever known,” Brody added in a press release. “They have been proven to treat an astounding variety of conditions, ranging from injuries to chronic, debilitating diseases.”
The Cures Act also has a number of provisions intended to speed approval of medical therapies. Chief among these is one that allows the FDA to use “real world evidence” in select reviews of treatments, and specifically pertains to new uses (or indications) of already approved drugs. Real world evidence covers such things as information from observational studies, patient input and anecdotal data. The act also allows for “data summaries,” rather than full clinical trial results, for the approval of new treatments.
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